Evidence map›Paper›PMID 39108094›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2024

Secondary failure of lentiviral vector gene therapy in a cerebral adrenoleukodystrophy patient with an ABCD1 whole-gene deletion.

Troy C Lund, Paul J Orchard, David R Nascene, Carina J King, Jennifer Braun, Stuti Thakkar, Willa Durose, Ilya Shestopalov, Himal Thakar, Ashish O Gupta

Abstract readCase Reports
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

4 citing papers in PubMed.

  1. Ex Vivo Gene and Cell Therapy in Hematopoietic Stem Cells.International journal of molecular sciences · 2025
    Review
  2. The curious case of AAV immunology.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  3. Review
  4. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

10 authors.

Troy C LundDepartment of Pediatrics, Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA. Electronic address: lundx072@umn.edu.
Paul J OrchardDepartment of Pediatrics, Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA.
David R NasceneDepartment of Diagnostic Radiology, University of Minnesota Medical Center, Minneapolis, MN, USA.
Carina J KingDepartment of Pediatrics, Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA.
Jennifer BraunDepartment of Pediatrics, Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA.
Stuti ThakkarDepartment of Pediatrics, Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA.
Willa DuroseDepartment of Pediatrics, Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA.
Ilya Shestopalovbluebird bio, Cambridge, MA, USA.
Himal Thakarbluebird bio, Cambridge, MA, USA.
Ashish O GuptaDepartment of Pediatrics, Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

A 9-year-old boy with adrenoleukodystrophy due to ABCD1 whole-gene deletion was diagnosed with active cerebral adrenoleukodystrophy characterized by demyelination and gadolinium enhancement on brain MRI. He underwent hematopoietic cell transplant (HCT) with autologous CD34

Indexed as

AdrenoleukodystrophyATP Binding Cassette Transporter, Subfamily D, Member 1Gene DeletionGenetic TherapyGenetic VectorsHematopoietic Stem Cell TransplantationLentivirusATP-Binding Cassette TransportersChildHumansMagnetic Resonance ImagingMaleABCD1 protein, humanATP-Binding Cassette TransportersATP Binding Cassette Transporter, Subfamily D, Member 1adrenoleukodystrophygene therapygraft failureimmune responselentivirustransplant

Identifiers

PMID39108094
PMCPMC11489540

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.